Trial Outcomes & Findings for Neoadjuvant Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF) in Cutaneous Stage L-lll Melanoma (NCT NCT02451488)

Results Overview

The Th1/Th2/Th3 Reverse transcription polymerase chain reaction (RT-qPCR) arrays will be used to quantify RNA expression of Th1 and Th2 messenger ribonucleic acids (mRNAs). Normalized gene expression was calculated from qRT-PCR results comparing GM-CSF and control subjects for both Th1-associated gene T-bet and Th2-associated gene GATA3 respectively. Fold change values are calculated by taking the normalized gene expression in GM-CSF treated group samples divided by the normalized gene expression in the control group samples.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

8 participants

Primary outcome timeframe

14 days post treatment

Results posted on

2020-02-10

Participant Flow

Participant milestones

Participant milestones
Measure	GM-CSF Patients will be treated with 14 days of GM-CSF self-administered subcutaneously daily for 14 days in a dose of 125 µg/m2 beginning on the day of enrollment. Patients will be instructed in the self-administration of GM-CSF and after they have demonstrated competency with the procedure, they will self-administer the treatment at home. Patients will undergo surgery within 1 day to 5 days after cessation of the GM-CSF therapy. GM-CSF: 14 days in a dose of 125 µg/m\^2	Standard of Care no neo-adjuvant therapy prior to surgical intervention Standard of Care: No neo-adjuvant therapy prior to surgical intervention
Overall Study STARTED	4	4
Overall Study COMPLETED	4	4
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	GM-CSF n=4 Participants Patients will be treated with 14 days of GM-CSF self-administered subcutaneously daily for 14 days in a dose of 125 µg/m2 beginning on the day of enrollment. Patients will be instructed in the self-administration of GM-CSF and after they have demonstrated competency with the procedure, they will self-administer the treatment at home. Patients will undergo surgery within 1 day to 5 days after cessation of the GM-CSF therapy. GM-CSF: 14 days in a dose of 125 µg/m\^2	Standard of Care n=4 Participants no neo-adjuvant therapy prior to surgical intervention Standard of Care: No neo-adjuvant therapy prior to surgical intervention	Total n=8 Participants Total of all reporting groups
Age, Continuous	54.5 years STANDARD_DEVIATION 14.3 • n=4 Participants	62.7 years STANDARD_DEVIATION 18.8 • n=4 Participants	58.6 years STANDARD_DEVIATION 16.1 • n=8 Participants
Sex: Female, Male Female	2 Participants n=4 Participants	2 Participants n=4 Participants	4 Participants n=8 Participants
Sex: Female, Male Male	2 Participants n=4 Participants	2 Participants n=4 Participants	4 Participants n=8 Participants
Race and Ethnicity Not Collected	—	—	0 Participants Race and Ethnicity were not collected from any participant.
Region of Enrollment United States	4 participants n=4 Participants	4 participants n=4 Participants	8 participants n=8 Participants

PRIMARY outcome

Timeframe: 14 days post treatment

Population: Three subjects were in in the GM-CSF group and three subjects were in the Standard of Care group. One subject in the GM-CSF group received GM-CSF but did not have tissue sample collected for research analysis. One subject in the Standard of Care group did not have a large enough tissue sample collected to be analyzed.

The Th1/Th2/Th3 Reverse transcription polymerase chain reaction (RT-qPCR) arrays will be used to quantify RNA expression of Th1 and Th2 messenger ribonucleic acids (mRNAs). Normalized gene expression was calculated from qRT-PCR results comparing GM-CSF and control subjects for both Th1-associated gene T-bet and Th2-associated gene GATA3 respectively. Fold change values are calculated by taking the normalized gene expression in GM-CSF treated group samples divided by the normalized gene expression in the control group samples.

Outcome measures

Outcome measures
Measure	GM-CSF n=3 Participants Patients will be treated with 14 days of GM-CSF self-administered subcutaneously daily for 14 days in a dose of 125 µg/m2 beginning on the day of enrollment. Patients will be instructed in the self-administration of GM-CSF and after they have demonstrated competency with the procedure, they will self-administer the treatment at home. Patients will undergo surgery within 1 day to 5 days after cessation of the GM-CSF therapy. GM-CSF: 14 days in a dose of 125 µg/m\^2	Standard of Care n=3 Participants no neo-adjuvant therapy prior to surgical intervention Standard of Care: No neo-adjuvant therapy prior to surgical intervention
Th1/Th2 Normalized Gene Expression T-bet (Th1)	1.029 fold change Interval 0.999 to 1.059	0.119 fold change Interval -1.506 to 1.654
Th1/Th2 Normalized Gene Expression GATA3 (Th2)	0.074 fold change Interval -0.041 to 0.279	0.192 fold change Interval 0.068 to 0.316

Adverse Events

GM-CSF

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Standard of Care

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr. James Jakub

Mayo Clinic

Phone: 507-284-3629

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place